;e newest member of this growing class of therapeutics is
ramucirumab (Cyramza). It was approved by the FDA for
the treatment of metastatic gastric (stomach) cancer and
cancer of the part of the esophagus that connects to the
stomach (gastroesophageal junction adenocarcinoma) in
April 2014. Patients with metastatic gastric cancer have a
very poor outlook; just 4 percent survive ;ve years ( 7). With
such a clear need for new treatment options, the fact that
ramucirumab extended overall survival for patients with
metastatic gastric cancer in phase III clinical trials ( 104,
105) provides patients with new hope.

Ramucirumab is also being tested in numerous clinical
trials as a potential treatment for other types of cancer.
Recent results from one of these trials showed that
ramucirumab signi;cantly prolonged survival for some
patients with the most deadly form of lung cancer, NSCLC
( 106). If these data result in an FDA approval, this will
provide more patients with new treatment options and
increase the return on prior investments in cancer research.

New Path to Approving Breast Cancer ;erapeutics

Breast cancer is the second leading cause of cancer-related
death for women in the United States ( 1). Studies have
shown that intervening early and aggressively can improve
survival for breast cancer patients who have a high risk of
recurrence. ;erefore, the FDA outlined a new path for
regulatory approval of breast cancer therapeutics in May
2012 ( 107) (see sidebar on New FDA Approach to Breast
Cancer Therapeutics).

In September 2013, pertuzumab (Perjeta) became the ;rst
therapeutic approved under this new regulatory path.

Pertuzumab is a therapeutic antibody that targets the HER2
protein. About one in every ;ve of the 235,030 cases of
breast cancer anticipated to be diagnosed in the United
States in 2014 will overexpress HER2 ( 1, 109).

;e FDA decision allows pertuzumab to be used as part of
a presurgery course of treatment for certain patients with
HER2-positive, early stage breast cancer. ;e decision was
based on clinical trial results showing that women who
received pertuzumab in addition to trastuzumab and the
traditional chemotherapy docetaxel before breast cancer
surgery were signi;cantly more likely to have no residual
invasive cancer detected in breast tissue and lymph nodes
removed during surgery compared with women who
received only trastuzumab and docetaxel ( 110).

It is important to note that these data are preliminary and
that we do not know for certain whether the pertuzumab-containing presurgery treatment will improve patients’

long-term outcomes, including survival. To determine this,
a large-scale clinical trial is ongoing and the results are
expected in 2016.

Treatment With Immunotherapeutics

A new approach to cancer treatment that has begun to
transform the lives of patients is immunotherapy.

Cancer immunotherapy refers to treatments that can
unleash the power of a patient’s immune system to
;ght cancer the way it ;ghts pathogens. Not all cancer
immunotherapies work in the same way (see sidebar on
How Immunotherapeutics Work, p. 65). As our scienti;c
understanding of the immune system and how it interacts
with cancer cells increases, we can expect to see novel
immunotherapies and new ways to use those that we
already have.

Given that some patients have remarkable and durable
responses following immunotherapy, this form of
cancer treatment holds incredible promise for the
future, potentially even cures for some patients. ;e
progress is very recent, and most experimental cancer
immunotherapies, which are the focus of the following
discussion, are still in clinical development and have,
therefore, not yet been approved by the FDA.